UF sees progress in potential new treatment for Type 1 diabetes

Dr. Michael Haller, an associate professor with the University of Florida Department of Pediatric Endocrinology, talks about his research dealing with Type 1 diabetes on Thursday. At back is type I diabetes patient Ryan Moss, 15.

Published: Thursday, June 19, 2014 at 4:35 p.m.

Last Modified: Thursday, June 19, 2014 at 4:35 p.m.

After several years of studies, University of Florida researchers say they have made progress in developing a potential new treatment for patients with Type 1 diabetes.

The treatment, which they have likened to a "cocktail therapy," involves two medications already approved by the Food and Drug Administration for different uses.

A recently concluded UF College of Medicine study showed that 17 Type 1 diabetes patients who went through treatment with the two drugs appeared to regain the ability to produce insulin, while eight patients who took a placebo did not.

"We got this synergistic effect," said Dr. Michael J. Haller, an associate professor of pediatric endocrinology at UF. "When you give the two together, it's synergy. It's one plus one equals 10."

In a statement, Mark Atkinson, a professor with the UF Department of Pathology, Immunology and Laboratory Medicine, and another researcher in the study said results so far have made him "cautiously optimistic."

Haller said UF researchers so far have overseen seven or eight years of studies on the potential therapy, beginning with mice and moving on to human patients. Larger trial studies will follow before the drugs could ever be used in treatment, he said.

But because the two medications already have FDA clearance for other uses, they potentially could be used to treat Type 1 diabetes without going through the lengthy FDA approval process for new drugs, Haller added.

Haller used a simplified analogy of cops and robbers to describe how the treatment works. First, Thymoglobulin, a drug initially developed for use in kidney transplant, kills some of the white blood cells in the immune system that are attacking the insulin-producing beta cells in the pancreas. Haller likened that to clearing out the bad guys, or robbers.

Next, Neulasta, a drug developed to treat cancer patients, is used to spur the production of new, healthy immune cells. Haller described those cells as the cops brought in for protection. Researchers said the study results indicate that the Thymoglobulin and Neulasta both worked as intended and that there was an increase in the insulin-producing beta cells in the pancreas.

He said the results were encouraging because the latest study was conducted on patients who had been diagnosed with Type 1 diabetes between four months and two years ago. That was significant because a lot of the beta cells often are wiped out in the first few months after a patient is diagnosed.

Haller said the eventual goal, years down the road, is developing a therapy that first uses an IV infusion of Thymoglobulin and then a Neulasta treatment once every two weeks for three months to greatly reduce or eliminate the need for some Type 1 diabetes patients to take insulin injections.

One potential candidate for such a treatment is Ryan Moss, 15, of Archer, who was diagnosed with Type 1 diabetes almost a year ago. Moss, who was at the UF Clinical Research Center on Thursday to have blood drawn in a separate ongoing test, said he has to take insulin shots five to six times a day.

When his blood sugar is low and he grows weak, Ryan knows he has to eat something.

At this point, it is not too burdensome dealing with the disease, Ryan said, because he has watched how his father, who also has Type 1 diabetes, handled it.

"Conceptually, he would be a great candidate for the therapy," Haller said of Ryan.

Type 1 diabetes, which previously was known as juvenile diabetes, makes up about 5 to 10 percent of the diagnosed cases in the country, according to the American Diabetes Association.

It's a chronic condition, typically diagnosed during childhood, where the pancreas no longer produces insulin or does not produce enough insulin.

The UF study, which researchers presented last weekend at the annual meeting of the American Diabetes Association in San Francisco, comes on the heels of a Centers for Disease Control and Prevention report stating that more than 29 million people in America now have diabetes.

The latest study was funded by the Helmsley Charitable Trust. Sanofi, the pharmaceutical company that holds the patent on Thymoglobulin, provided that drug free of charge.

<p>After several years of studies, University of Florida researchers say they have made progress in developing a potential new treatment for patients with Type 1 diabetes.</p><p>The treatment, which they have likened to a "cocktail therapy," involves two medications already approved by the Food and Drug Administration for different uses.</p><p>A recently concluded UF College of Medicine study showed that 17 Type 1 diabetes patients who went through treatment with the two drugs appeared to regain the ability to produce insulin, while eight patients who took a placebo did not.</p><p>"We got this synergistic effect," said Dr. Michael J. Haller, an associate professor of pediatric endocrinology at UF. "When you give the two together, it's synergy. It's one plus one equals 10."</p><p>In a statement, Mark Atkinson, a professor with the UF Department of Pathology, Immunology and Laboratory Medicine, and another researcher in the study said results so far have made him "cautiously optimistic."</p><p>Haller said UF researchers so far have overseen seven or eight years of studies on the potential therapy, beginning with mice and moving on to human patients. Larger trial studies will follow before the drugs could ever be used in treatment, he said.</p><p>But because the two medications already have FDA clearance for other uses, they potentially could be used to treat Type 1 diabetes without going through the lengthy FDA approval process for new drugs, Haller added.</p><p>Haller used a simplified analogy of cops and robbers to describe how the treatment works. First, Thymoglobulin, a drug initially developed for use in kidney transplant, kills some of the white blood cells in the immune system that are attacking the insulin-producing beta cells in the pancreas. Haller likened that to clearing out the bad guys, or robbers.</p><p>Next, Neulasta, a drug developed to treat cancer patients, is used to spur the production of new, healthy immune cells. Haller described those cells as the cops brought in for protection. Researchers said the study results indicate that the Thymoglobulin and Neulasta both worked as intended and that there was an increase in the insulin-producing beta cells in the pancreas.</p><p>He said the results were encouraging because the latest study was conducted on patients who had been diagnosed with Type 1 diabetes between four months and two years ago. That was significant because a lot of the beta cells often are wiped out in the first few months after a patient is diagnosed.</p><p>Haller said the eventual goal, years down the road, is developing a therapy that first uses an IV infusion of Thymoglobulin and then a Neulasta treatment once every two weeks for three months to greatly reduce or eliminate the need for some Type 1 diabetes patients to take insulin injections.</p><p>One potential candidate for such a treatment is Ryan Moss, 15, of Archer, who was diagnosed with Type 1 diabetes almost a year ago. Moss, who was at the UF Clinical Research Center on Thursday to have blood drawn in a separate ongoing test, said he has to take insulin shots five to six times a day.</p><p>When his blood sugar is low and he grows weak, Ryan knows he has to eat something.</p><p>At this point, it is not too burdensome dealing with the disease, Ryan said, because he has watched how his father, who also has Type 1 diabetes, handled it.</p><p>"Conceptually, he would be a great candidate for the therapy," Haller said of Ryan.</p><p>Type 1 diabetes, which previously was known as juvenile diabetes, makes up about 5 to 10 percent of the diagnosed cases in the country, according to the American Diabetes Association.</p><p>It's a chronic condition, typically diagnosed during childhood, where the pancreas no longer produces insulin or does not produce enough insulin.</p><p>The UF study, which researchers presented last weekend at the annual meeting of the American Diabetes Association in San Francisco, comes on the heels of a Centers for Disease Control and Prevention report stating that more than 29 million people in America now have diabetes.</p><p>The latest study was funded by the Helmsley Charitable Trust. Sanofi, the pharmaceutical company that holds the patent on Thymoglobulin, provided that drug free of charge.</p>